PROJECT SUMMARY ? GCD CORE The Gene and Cell Delivery (GCD) core will be a new component of the UCLA vision research core grant. The establishment of this core is in response to the research demands by UCLA vision scientists to deliver reagents to the retina, RPE, and other ocular tissues for the purpose of investigating basic mechanisms and preclinical testing of therapeutic potentials. The GCD core is consist of a biosafety level 2 (BSL2) viral vector production facility and a facility for have a technical staff with molecular biology, cell culture, recombinant virus preparation expertise. Ideally, the GCD staff will also have experiences of operating injection instrument and can teach members of individual laboratories to perform subretinal and intravitreal delivery in neonatal and adult rodents. The GCD core will provide support for the production of recombinant viral vectors, mainly rAAVs and lentiviruses. A website will be set up to provide available viral vector backbone information and for individual labs to submit requests. Under the supervision of the core component director Dr. X-J Yang, the GCD staff will use viral vector constructs and other necessary reagents provided by individual investigator labs to perform transfection, harvesting, viral concentration, and tittering procedures. The viral stocks produced by the GCD core will be tested by labs of individual investigators. The GCD core is equipped with necessary microscopes and micromanipulators to perform intraocular injections in a designed BSL2 animal facility. The reagents to be delivered using these equipment/instruments can range from nucleic acids, protein conjugates, nanoparticles, recombinant viruses, small molecules, and human stem cell-derived cells. The same facility can house post-operative mice deemed to need BSL2 containment. The GCD core is expected to facilitate basic and preclinical research supported by NIH/NEI on UCLA campus by providing effective tools and by enabling intraocular delivery in animal models. 1